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Treatment Effect of Tamoxifen on Patients With DMD

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ClinicalTrials.gov Identifier: NCT02835079
Recruitment Status : Completed
First Posted : July 15, 2016
Last Update Posted : July 25, 2022
Information provided by (Responsible Party):
Talia Dor, Hadassah Medical Organization

Brief Summary:

Duchenne muscular dystrophy (DMD) is a progressive devastating disease that affects mainly boys, with an incidence of about 1:3,500 live births. The pathology of DMD is a result of non-repaired muscle damage that leads to muscle-tissue replacement by scar tissue, a process known as fibrosis. Currently, there is no effective treatment for the disease. The only therapy offered to these boys are steroids which slightly delayed the disease progression. The boys lose their ability to walk at around the age of 12, and die in the 4th decade of life from severe heart and lung problems.

In this study investigators will test the efficacy of Tamoxifen treatment in ambulatory DMD boys. Tamoxifen is a drug used for palliative treatment of breast cancer patients and has an outstanding safety profile. In addition, Tamoxifen was tested in the past in boys, for other pediatric indications, and showed an excellent safety with no side effects.

Tamoxifen is being tested in this study, as a therapy for DMD, for the following reasons:

(i) it was shown to have anti-fibrotic effect in multiple in-vivo systems; (ii) it assists in the repair of damaged muscles.

In other words, Tamoxifen is expected to have a synergistic effect on DMD patients, due to its dual mechanism of action. Indeed, Tamoxifen was shown to have significant beneficial effects in the mdx mouse model of DMD. Also, a small compassionate cohort of 3 boys, treated for 6 months with Tamoxifen, yielded very encouraging results.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Tamoxifen Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment Effect of Tamoxifen on Patients With DMD
Actual Study Start Date : November 2016
Actual Primary Completion Date : November 2020
Actual Study Completion Date : November 2020

Arm Intervention/treatment
open label study
one arm open label study
Drug: Tamoxifen

Primary Outcome Measures :
  1. 6-minute walk distance (6MWD) [ Time Frame: The six minute walk distance will be tested during the 36 months of the trial. For the first 12 months, the 6 minute walk test will be tested every 3 months and for the follow up period of 24 months,will be done every 6 months. ]
    PT evaluation

Secondary Outcome Measures :
  1. North Star assesment(NSAA) [ Time Frame: The NSAA will be tested during the 36 months of the trial. For the first 12 months, the NSAA will be tested every 3 months and for the follow up period of 24 months,will be done every 6 months. ]
    PT evaluation

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 16 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Ambulatory

Exclusion Criteria:

  • Non Ambulatory
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Talia Dor, MD, Hadassah Medical Organization
ClinicalTrials.gov Identifier: NCT02835079    
Other Study ID Numbers: DMDTAM001-HMO-CTIL
First Posted: July 15, 2016    Key Record Dates
Last Update Posted: July 25, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Talia Dor, Hadassah Medical Organization:
Duchenne Tamoxifen
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Estrogen Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Selective Estrogen Receptor Modulators
Estrogen Receptor Modulators
Bone Density Conservation Agents